Investigators at the Institute for Research in Biomedicine (IRB Barcelona) have unraveled how and why the absence of a neuronal microexon in cytoplasmic polyadenylation element-binding 4 (CPEB4) gives rise to autism. In 2018, investigators from IRB, co-led by Raúl Méndez, identified the overt correlation between defects in CPEB4 and the onset of autism. However, the previous work did not provide the molecular mechanism explaining the correlation.
Investigators at the Institute for Research in Biomedicine (IRB Barcelona) have unraveled how and why the absence of a neuronal microexon in cytoplasmic polyadenylation element-binding 4 (CPEB4) gives rise to autism. In 2018, investigators from IRB, co-led by Raúl Méndez, identified the overt correlation between defects in CPEB4 and the onset of autism. However, the previous work did not provide the molecular mechanism explaining the correlation.
To address the existing challenges with CAR T-cell therapy, scientists at Umoja Biopharma Inc. developed the Vivovec platform, which is an off-the-shelf surface-engineered lentiviral vector (LVV) drug product designed to generate CAR T cells in vivo without lymphodepletion by selectively binding, activating and transducing T cells.
Peptidream Inc. has announced a new peptide radiopharmaceutical development candidate, PD-29875, a novel first-in-class highly selective macrocyclic peptide-radioisotope conjugate against Claudin 18.2 (CLDN18.2).
Opna Bio AG has presented promising data regarding their EP300/CBP inhibitor OPN-6602 for the treatment of multiple myeloma (MM). The inhibition of EP300/CBP causes cell cycle arrest and apoptosis in MM cells due to suppression of interferon regulatory factor 4 (IRF4) and MYC repression.
SK Bioscience has received approval from the Human Research Ethics Committee in Australia for a phase I/II trial of its mRNA-based Japanese encephalitis vaccine candidate, GBP-560.
Elevation Oncology Inc. has nominated EO-1022 as its HER3 antibody-drug conjugate (ADC) development candidate for the treatment of solid tumors that express HER3.
Baylor College of Medicine has disclosed proteolysis targeting chimeras (PROTACs) comprising cereblon (CRBN) ligands coupled to Bruton tyrosine kinase (BTK)-, DNA-binding protein Ikaros (IKZF1)- and Zinc finger protein Aiolos (IKZF3)-targeting moieties through a linker.
Karen Carey, BioWorld’s managing editor and chief analyst, and the managing editor of Bioworld Science, Anette Breindl, join this episode of the BioWorld Insider podcast to talk about the business and the science of women’s health.
Epileptic encephalopathy due to mutations in the STXBP1 gene, also known as genetic epilepsy, is a rare disease characterized by intellectual disability, speech and motor impairment and behavioral issues, among others, that affects 1 in every 30,000 newborns and which has no approved therapies to...
Researchers from State University of New Jersey (Rutgers) presented preclinical data for PW-507, a sigma-1 receptor antagonist being evaluated for the treatment of binge eating disorder.
Pathogenic variants in the KCNT1 gene, which encodes potassium channel subfamily T member 1, cause a severe childhood developmental epileptic encephalopathy.
Gynecological cancers with amplifications in the CCNE1 gene, which encodes cyclin E1, usually exhibit resistance to standard therapies. Since cyclin-dependent kinase 2 (CDK2) is the primary partner of cyclin E, CDK2 inhibitors represent a potentially effective treatment strategy for these...
Previous research suggested that gram-negative bacteria may hold potential for cancer immunotherapy because they contain agonists for multiple immune receptors.
Researchers from Inovio Pharmaceuticals Inc. described the protective effect exerted by INO-4500, a DNA vaccine produced using the Syncon technology, against lethal Lassa virus in nonhuman primates.
Orthogon Therapeutics LLC has closed an oversubscribed financing round, with funding to be used to accelerate the development of its first-in-class treatments for managing BK virus reactivation in transplant patients.
Researchers from Maplight Therapeutics Inc. presented preclinical data for the investigational muscarinic M1/M4 receptor agonist ML-007, being evaluated for the treatment of neuropsychiatric disorders, such as schizophrenia and Alzheimer’s disease (AD). The activity of ML-007 was compared to that...
Researchers from the University of California San Francisco (UCSF) have successfully replicated the design of regulatory T cells, achieving local targeted immune suppression and protection from CAR T-cell cytotoxicity. Many of the treatments used so far in the context of inflammatory and autoimmune...
Dewpoint Therapeutics Inc. and Mitsubishi Tanabe Pharma Corp. have entered a research collaboration worth up to $480 million to advance Dewpoint’s novel TDP-43 small-molecule condensate modulator for amyotrophic lateral sclerosis. Under terms of the deal, Boston-based Dewpoint will receive an...
Researchers from Bright Minds Biosciences Inc. presented data on the mechanism of action of BMB-101, a novel scaffold 5-HT2C Gq-protein biased designed to treat drug-resistant neurological disorders.
Researchers at the University of Rochester have described a neuroimaging-based biomarker that could identify individuals with early psychosis, and improved their identification when it was added to a standard neurocognitive diagnostic test. In a group of roughly 160 participants in the Human...
WD repeat domain 83 opposite strand (WDR83OS) encodes the 106-aa (amino acid) protein Asterix, which is a binding partner for CCDC47. More specifically, Asterix heterodimerizes with CCDC47 to form the protein associated with ER translocon (PAT) complex that specifically chaperones large proteins...
Amgen Inc. has reported new 5,6- and 6,6-fused bicyclic alcohols and ethers acting as 15-hydroxyprostaglandin dehydrogenase (15-PGDH) inhibitors. They are reported to be useful for the treatment of gastrointestinal disorders.
Recently presented preclinical data show that EA Pharma Co. Ltd.'s EA-3571 is a highly potent dual inhibitor of enteropeptidase and trypsin with luminal action, resulting in potential anti-insulin resistance and fat-burning properties.
The enzyme aminocarboxymuconate semialdehyde decarboxylase (ACMSD) is a regulator of de novo NAD+ synthesis and is reduced in patients with advanced liver disease.
Rectify Pharmaceuticals Inc. has conducted preclinical testing on RTY-694, an oral dual-targeted positive functional modulator for use in the treatment of hepatobiliary disorders.
Breakthrough medicines, billion-dollar deals, spectacular clinical successes and crushing failures all play a part in biopharma’s dynamic story. Developers make scientific advancements with the potential to change everything, only to face regulatory conundrums and ever-fluctuating markets. BioWorld tracks key events in the fast-moving sector every business day. Now, the BioWorld Insider podcast lets you hear directly from the movers and shakers whose collective work is changing how we all live. Join us each week for a new conversation.